Translational Notes: TRNDing toward translation

 

Table 1. TRND partnerships. List of NIH Therapeutics for Rare & Neglected Diseases (TRND) partnerships as of Oct. 6, 2011.

Source: NIH Center for Translational Therapeutics (NCTT)

Pilot projects

Company/institutional partners

Compound

Target/mechanism

Indication

AesRx LLC

Aes-103

Small molecule that binds sickle hemoglobin

Sickle cell disease

New Zealand Pharmaceuticals Ltd./National Human Genome Research Institute

N-Acetyl-d-mannosamine (ManNAc)

Corrects sialic acid deficiency

Hereditary inclusion body myopathy (hIBM)

Leukemia & Lymphoma Society/University of Kansas Medical Center

Auranofin

Selectively kills chronic lymphocytic leukemia cells

Chronic lymphocytic leukemia (CLL)

Various Niemann-Pick disease type C foundations/Washington University in St. Louis/Albert Einstein College of Medicine of Yeshiva University

2-Hydroxypropyl-b-cyclodextrin (HPBCD)

Decreases cholesterol and sphingolipid storage

Niemann-Pick disease type C1 (NPC1)

NIH Chemical Genomics Center/Rush University Medical Center/Yale University

Oxadiazole 2-oxides

Inhibits schistosome redox pathways

Schistosomiasis and hookworm disease

First round of solicited projects

Company/institutional partners

Compound

Target/mechanism

Indication

Afraxis Inc.

Not applicable

p21 Protein (Cdc42 Rac)-activated kinase 1 (PAK1) inhibitor

Fragile X syndrome

ReveraGen BioPharma Inc.

VBP15

Modified glucocorticoid with reduced side effects

Duchenne muscular dystrophy (DMD)

Viamet Pharmaceuticals Inc.

VT-1129

Cytochrome P450 C-14 a-demethylase (CYP51) inhibitor

Cryptococcal meningitis

National Human Genome Research Institute

Not applicable

Inhibitor of core binding factor (CBF) protein interactions

CBF-driven acute myelogenous leukemia (AML) and acute lymphoblastic leukemia (ALL)